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New treatment option for Refractory Idiopathic Aplastic Anemia
Official title Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide in Patients With Acquired Refractory Aplastic Anemia or in Relapse After Immunosuppression
ClinicalTrials.gov ID: NCT05126849
What this study is testing
- What it's testing
- Outcomes for patients with severe aplastic anemia (SAA) who are refractory to first-line immunosuppressive therapy (IST) and who lack a matched unrelated donor (MUD) remain poor. Recently, the use of eltrombopag (ELT) has shown blood count improvements in 40% of these patients.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 3 to 35
You may be able to join if
- Aged from 3 to 35 years old
- Suffering from refractory acquired idiopathic aplastic anemia (at least one course of immunosuppression with anti-thymocyte globulin)
- Absence of geno-identical donor or 10/10 matched donor
- With identification of a haploidentical donor (brother, sister, parents, adult children or cousin)
- Absence of donor specific antibody detected in the patient with a MFI ≥ 1500 (antibodies directed towards the distinct haplotype between donor and...
You likely can't join if
- With morphologic evidence of clonal evolution (patients with isolated bone marrow cytogenetic abnormalities are also eligible excepted chromosome 7...
- With uncontrolled infection
- With seropositivity for HIV or HTLV-1 (Human T cell Leukemia) or active hepatitis B or C defined by a positive PCR (polymerase chain reaction) HBV...
- Cancer in the last 5 years (except basal cell carcinoma of the skin or "in situ" carcinoma of the cervix)
- Pregnant (βHCG positive) or breast-feeding.
- Who received live attenuated vaccine within 2 months before transplantation and during the research
See the full eligibility criteria
- Aged from 3 to 35 years old
- Suffering from refractory acquired idiopathic aplastic anemia (at least one course of immunosuppression with anti-thymocyte globulin)
- Absence of geno-identical donor or 10/10 matched donor
- With identification of a haploidentical donor (brother, sister, parents, adult children or cousin)
- Absence of donor specific antibody detected in the patient with a MFI ≥ 1500 (antibodies directed towards the distinct haplotype between donor and recipient)
- With usual criteria for HSCT :
- ECOG(Eastern Cooperative Oncology Group) ≤ 2
- No severe and uncontrolled infection
- Cardiac function compatible with high dose of cyclophosphamide
- Adequate organ function: ASAT(aspartate transaminase) and ALAT (alanine aminotransferase) ≤ 2.5 N (the norm), total bilirubin ≤ 2N, creatinine \< 150 μmol/L
- With health insurance coverage
- Contraception methods must be prescribed during all the duration of the research. Women and men of childbearing age must use contraceptive methods within 12 months and 6 months after the last dose of cyclophosphamide...
- Having signed a written informed consent (2 parents for patients aged less than 18)
- With morphologic evidence of clonal evolution (patients with isolated bone marrow cytogenetic abnormalities are also eligible excepted chromosome 7 abnormalities and complex karyotype)
- With uncontrolled infection
- With seropositivity for HIV or HTLV-1 (Human T cell Leukemia) or active hepatitis B or C defined by a positive PCR (polymerase chain reaction) HBV (hepatitis B virus) or HCV (hepatitis C virus) and associated hepatic...
- Cancer in the last 5 years (except basal cell carcinoma of the skin or "in situ" carcinoma of the cervix)
- Pregnant (βHCG positive) or breast-feeding.
- Who received live attenuated vaccine within 2 months before transplantation and during the research
- Uncontrolled coronary insufficiency, recent myocardial infarction \<6 month, current manifestations of heart failure, uncontrolled cardiac rhythm disorders, ventricular ejection fraction \<50%
- With heart failure according to NYHA (New York Heart Association) (II or more)
- Preexisting acute hemorrhagic cystitis
- Renal failure with creatinine clearance \<30ml / min
- With urinary tract obstruction
- Who receive the following treatments Phenytoin, Pentostatin, inhibitor of adenoside)
- Who have any debilitating medical or psychiatric illness, which preclude understanding the inform consent as well as optimal treatment and follow-up
- Under tutorship or curatorship
The study team makes the final eligibility decision.
Where it's taking place
- Amiens, France
- Angers, France
- Besançon, France
- Bordeaux, France
- Caen, France
- Clamart, France
- Clermont-Ferrand, France
- Créteil, France
- Grenoble, France
- Lille, France
- Limoges, France
- Lyon, France
- Marseille, France
- Montpellier, France
- Nancy, France
- Nantes, France
- Nice, France
- Paris, France
- Poitiers, France
- Rennes, France
+ 4 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 3 years to 35 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Amiens, France; Angers, France; Besançon, France; Bordeaux, France; Caen, France; Clamart, France and 18 more location(s). Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.